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Open Access
  • © Michaël Hocquemiller
  • , et al.
  • 2022

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

  • Michaël Hocquemiller 1
  • Laura Giersch 1
  • Mickael Audrain 2
  • Samantha Parker 1
  • Nathalie Cartier 3
  • 1 - Lysogene - Neuilly sur Seine France
  • 2 - Université Paris Descartes - Paris France
  • 3 - INSERM UMR1169 - Université Paris-Sud - Université Paris-Saclay - Orsay - France


Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and efficiency in transducing a wide range of cell types. The purpose of this review is to describe the most notable advancements in preclinical and clinical research on AAV-based CNS gene therapy and to discuss prospects for future development based on a new generation of vectors and delivery.